This historic decision would make available on the NHS the first ever drug to treat an underlying genetic cause of Duchenne muscular dystrophy.
In order to give time for NHS England and the drug company, PTC Therapeutics, to agree an final acceptable price, NICE has set July as the date where its recommendation will become final. We’ll then be pushing NHS England to implement this decision in order to get the drug to boys in clinic as quickly as possible.
A new drug is giving hope to boys in Europe with Duchenne Muscular Dystrophy, but it's not yet been made available on the NHS in the UK. A petition to change that has been signed by thousands of people.
A nine-year-old boy with a rare muscle wasting disease has made a personal plea to the Prime Minister to make the drug he needs available on the NHS. Archie Hill handed in a petition at Number 10. He was joined in Downing Street by families from across the South who have youngsters suffering from the same condition. Phil Hornby reports (video courtesy of Meridian News )
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Nine year-old Archie Hill, of Gerrards Cross, Buckinghamshire, who has muscular dystrophy is handing in his personal plea to Prime Minister David Cameron to help him.
A petition of 22,000 signatures will be presented at 10 Downing Street by families waiting for access to Translarna, the first ever drug tackling the causes of Duchenne muscular dystrophy.
In a statement NHS England says:“Patient groups have asked the NHS to consult with the public about priorities for new services and treatments to be funded from April, and that consultation begins next week.
"Meanwhile patients whose doctors say they have an urgent need for a new treatment can ask medical experts to fast track their case.”
A petition signed by more than 23,000 people is to be presented to David Cameron, urging him to get the drug Translarna approved for boys who have a certain kind of Duchenne Muscular Dystrophy.
The drug was approved in Europe in August.
Families of children with the condition claim they are being left to suffer due to NHS administrative delays.
"I think you might be interested to see the update set out in my note (with a photo) which I sent to the staff team following the success of the Parliamentary work and the media push yesterday, including a meeting with David Cameron in Downing Street. We had an exceptionally good day and the team is now completing the follow up actions to take this forward.